Education Advocacy Communications | 23 November 2018

Statement from Retina International on the EMA Decision to Approve Luxturna


Statement from Retina International on the EMA Decision to Approve Luxturna

Zurich, November 23rd, 2018 - Retina International, the global umbrella group of 43 patient-led organisations on all continents supporting research into retinal degenerative diseases, including Inherited Retinal Dystrophies - IRDs, welcomes today’s announcement that the European Medicines Agency (EMA) has approved voretigene neparvovec (tradename: Luxturna) a gene therapy for the treatment of biallelic RPE65 a form of Leber’s Congenital Amaurosis, LCA.

Luxturna has been approved for use in children and adults with vision loss due to inherited retinal diseases (IRD) caused by mutations in both copies (biallelic) of the RPE65 gene and who have sufficient viable retinal cells.

The RPE65 gene works by providing instructions to make an enzyme (a protein that facilitates chemical reactions) that is essential for normal vision. Individuals with biallelic RPE65 mutation-associated retinal dystrophy experience progressive deterioration in their vision over time. Luxturna works by delivering a normal copy of the RPE65 gene directly to retinal cells via a sub retinal injection. These retinal cells then produce the normal protein that converts light to an electrical signal in the retina to restore vision. It is administered as a one-time treatment by a specialist eye surgeon.

While Luxturna has now been approved by both the EMA in Europe and the FDA in the United States as safe and effective, and although it is now on the market in the United States, this does not mean immediate access throughout Europe. The next stage on the journey to access will be at a local level. National agencies in certain countries will now decide whether to approve the therapy for reimbursement as part of a Health Technology Assessment (HTA) process or similar.

President of Retina International, Ms Christina Fasser said of the approval ‘Retina International welcomes today’s announcement that the EMA has approved Luxturna for the treatment of Biallelic LCA, RPE65. This is the first treatment for a form of Inherited Retinal Dystrophy and is a landmark day for the retina community at large. However, there is much work to be done before access to this potentially curative treatment is possible for eligible patients in Europe. Many European countries will now conduct a Health Technology Assessment or a HTA on this product to evaluate the cost effectiveness of the therapy and this may take some time’. She continued ‘As the umbrella group representing the voice of patients living with inherited retinal degenerative diseases (IRDs) all over the world, Retina International asks those national agencies to understand that these conditions are degenerative and so time is of the essence in gaining access to treatment that can be life changing for those affected, their families and all those who care for them’.


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Christina Fasser,

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